Gene

Cell biology

Haematopoiesis

Molecular biology

Biology

Transgene

Cas9

Genome editing

Transplantation

Stem cell

CRISPR

Fetus

Hemoglobin

Cancer research

Biochemistry

Computational biology

Globin

Hematopoietic stem cell

Genetics

Xenotransplantation

Progenitor cell

Internal medicine

Medicine

Fetal hemoglobin

Pregnancy

Blood Advances

Key Points Cas9 editing of the γ-globin gene promoters in hematopoietic stem cells (HSCs) increases red cell HbF by ≤40%. No deleterious effects on hematopoiesis or off-target mutations were detected 16 weeks after xenotransplantation of edited HSCs.

Genome editing of HBG1 and HBG2 to induce fetal hemoglobin